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Pediatric Nephrology

Erschienen in:

01.04.2016 | Original Article

Anti-interleukin 1 treatment in secondary amyloidosis associated with autoinflammatory diseases

verfasst von: Rezan Topaloglu, Ezgi Deniz Batu, Diclehan Orhan, Seza Ozen, Nesrin Besbas

Erschienen in: Pediatric Nephrology | Ausgabe 4/2016

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Abstract

Background

Amyloidosis may complicate autoinflammatory diseases (AID). We aimed to evaluate the renal biopsy findings, and clinical and laboratory parameters in patients with AID-associated amyloidosis who have responded to anti-interleukin 1(IL1) treatment.

Methods

Two children with systemic juvenile idiopathic arthritis and one with cryopyrin-associated periodic syndrome diagnosed as having reactive amyloidosis were treated with anti-IL1 drugs. The renal histopathological findings at the time of diagnosis of amyloidosis and after the onset of anti-IL1 were evaluated according to the amyloid scoring/grading system.

Results

The median age of disease onset and diagnosis of amyloidosis were 3 and 12 years, respectively. Anakinra was started in all; however, anakinra caused a local cutaneous reaction in one, thus canakinumab was commenced. Proteinuria improved in all. Control renal biopsies were performed a median of 3 years after the first biopsies. The renal amyloid prognostic score did not improve in patient 1, and progressed in patients 2 and 3. The renal amyloid grade progressed in patient 2.

Conclusions

This is the first series demonstrating progression of renal tissue damage after the improvement of proteinuria with anti-IL 1 in AID-associated amyloidosis. Anti-IL1 drugs are important to prevent further amyloid accumulation; however, new treatment strategies are needed to target the amyloid deposits.

Merlini G, Bellotti V (2003) Molecular mechanisms of amyloidosis. N Engl J Med 349:583–596CrossRefPubMed

Real de Asua D, Costa R, Galvan JM, Filigheddu MT, Trujillo D, Cadinanos J (2014) Systemic AA amyloidosis: epidemiology, diagnosis, and management. J Clin Epidemiol 6:369–377CrossRef

Lachmann HJ, Goodman HJ, Gilbertson JA, Gallimore JR, Sabin CA, Gillmore JD, Hawkins PN (2007) Natural history and outcome in systemic AA amyloidosis. N Engl J Med 356:2361–2371CrossRefPubMed

David J (1991) Amyloidosis in juvenile chronic arthritis. Clin Exp Rheumatol 9:73–78PubMed

Akpolat T, Ozkaya O, Ozen S (2012) Homozygous M694V as a risk factor for amyloidosis in Turkish FMF patients. Gene 492:285–289CrossRefPubMed

Dixit R, Gupta R, Dave L, Prasad N, Sharma S (2009) Clinical profile of patients having pulmonary tuberculosis and renal amyloidosis. Lung India 26:41–45PubMedCentralCrossRefPubMed

Hoffman HM, Mueller JL, Broide DH, Wanderer AA, Kolodner RD (2001) Mutation of a new gene encoding a putative pyrin-like protein causes familial cold autoinflammatory syndrome and Muckle–Wells syndrome. Nat Genet 29:301–305PubMedCentralCrossRefPubMed

Petty RE, Southwood TR, Baum J, Bhettay E, Glass DN, Manners P, Maldonado-Cocco J, Suarez-Almazor M, Orozco-Alcala J, Prieur AM (1998) Revision of the proposed classification criteria for juvenile idiopathic arthritis: Durban, 1997. J Rheumatol 25:1991–1994PubMed

Petty RE, Southwood TR, Manners P, Baum J, Glass DN, Goldenberg J, He X, Maldonado-Cocco J, Orozco-Alcala J, Prieur AM, Suarez-Almazor ME, Woo P, International League of Associations for Rheumatology (2004) International League of Associations for Rheumatology classification of juvenile idiopathic arthritis: second revision, Edmonton, 2001. J Rheumatol 31:390–392PubMed

10.

Thierry S, Fautrel B, Lemelle I, Guillemin F (2014) Prevalence and incidence of juvenile idiopathic arthritis: a systematic review. Joint Bone Spine 81:112–117CrossRefPubMed

11.

Woerner A, von Scheven-Gete A, Cimaz R, Hofer M (2015) Complications of systemic juvenile idiopathic arthritis: risk factors and management recommendations. Expert Rev Clin Immunol 11:575–588CrossRefPubMed

12.

Ozen S, Bilginer Y (2014) A clinical guide to autoinflammatory diseases: familial Mediterranean fever and next-of-kin. Nat Rev Rheumatol 10:135–147CrossRefPubMed

13.

Giat E, Lidar M (2014) Cryopyrin-associated periodic syndrome. Isr Med Assoc J 16:659–661PubMed

14.

Zhao Y, Wallace C (2014) Judicious use of biologicals in juvenile idiopathic arthritis. Curr Rheumatol Rep 16:454CrossRefPubMed

15.

Duarte C, Gomes C, Correia AJ, Salgado M (2006) Renal amyloidosis: an uncommon complication of juvenile idiopathic arthritis. Clin Rheumatol 25:548–549CrossRefPubMed

16.

Ter Haar N, Lachmann H, Ozen S, Woo P, Uziel Y, Modesto C, Kone-Paut I, Cantarini L, Insalaco A, Neven B, Hofer M, Rigante D, Al-Mayouf S, Touitou I, Gallizzi R, Papadopoulou-Alataki E, Martino S, Kuemmerle-Deschner J, Obici L, Iagaru N, Simon A, Nielsen S, Martini A, Ruperto N, Gattorno M, Frenkel J, Paediatric Rheumatology International Trials Organisation (PRINTO) and the Eurofever/Eurotraps Projects (2013) Treatment of autoinflammatory diseases: results from the Eurofever Registry and a literature review. Ann Rheum Dis 72:678–685CrossRefPubMed

17.

Stoll ML, Cron RQ (2014) Treatment of juvenile idiopathic arthritis: a revolution in care. Pediatr Rheumatol Online J 12:13PubMedCentralCrossRefPubMed

18.

Urieli-Shoval S, Linke RP, Matzner Y (2000) Expression and function of serum amyloid A, a major acute-phase protein, in normal and disease states. Curr Opin Hematol 7:64–69CrossRefPubMed

19.

Obici L, Perfetti V, Palladini G, Moratti R, Merlini G (2005) Clinical aspects of systemic amyloid diseases. Biochim Biophys Acta 1753:11–22CrossRefPubMed

20.

Gillmore JD, Lovat LB, Persey MR, Pepys MB, Hawkins PN (2001) Amyloid load and clinical outcome in AA amyloidosis in relation to circulating concentration of serum amyloid A protein. Lancet 358:24–29CrossRefPubMed

21.

Sen S, Sarsik B (2010) A proposed histopathologic classification, scoring, and grading system for renal amyloidosis: standardization of renal amyloid biopsy report. Arch Pathol Lab Med 134:532–544PubMed

22.

Sayed RH, Hawkins PN, Lachmann HJ (2015) Emerging treatments for amyloidosis. Kidney Int 87:516–526CrossRefPubMed

23.

Dinarello CA, Simon A, van der Meer JW (2012) Treating inflammation by blocking interleukin-1 in a broad spectrum of diseases. Nat Rev Drug Discov 11:633–652PubMedCentralCrossRefPubMed

24.

Park H, Bourla AB, Kastner DL, Colbert RA, Siegel RM (2012) Lighting the fires within: the cell biology of autoinflammatory diseases. Nat Rev Immunol 12:570–580PubMedCentralCrossRefPubMed

25.

DeWitt EM, Kimura Y, Beukelman T, Nigrovic PA, Onel K, Prahalad S, Schneider R, Stoll ML, Angeles-Han S, Milojevic D, Schikler KN, Vehe RK, Weiss JE, Weiss P, Ilowite NT, Wallace CA, Juvenile Idiopathic Arthritis Disease-specific Research Committee of Childhood Arthritis Rheumatology and Research Alliance (2012) Consensus treatment plans for new-onset systemic juvenile idiopathic arthritis. Arthritis Care Res 64:1001–1010

26.

Yokota S, Imagawa T, Mori M, Miyamae T, Aihara Y, Takei S, Iwata N, Umebayashi H, Murata T, Miyoshi M, Tomiita M, Nishimoto N, Kishimoto T (2008) Efficacy and safety of tocilizumab in patients with systemic-onset juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled, withdrawal phase III trial. Lancet 371:998–1006CrossRefPubMed

27.

Aoki C, Inaba Y, Choe H, Kaneko U, Hara R, Miyamae T, Imagawa T, Mori M, Oba MS, Yokota S, Saito T (2014) Discrepancy between clinical and radiological responses to tocilizumab treatment in patients with systemic-onset juvenile idiopathic arthritis. J Rheumatol 41:1171–1177CrossRefPubMed

28.

Gattorno M, Piccini A, Lasiglie D, Tassi S, Brisca G, Carta S, Delfino L, Ferlito F, Pelagatti MA, Caroli F, Buoncompagni A, Viola S, Loy A, Sironi M, Vecchi A, Ravelli A, Martini A, Rubartelli A (2008) The pattern of response to anti-interleukin-1 treatment distinguishes two subsets of patients with systemic-onset juvenile idiopathic arthritis. Arthritis Rheum 58:1505–1515CrossRefPubMed

29.

Nigrovic PA, Mannion M, Prince FH, Zeft A, Rabinovich CE, van Rossum MA, Cortis E, Pardeo M, Miettunen PM, Janow G, Birmingham J, Eggebeen A, Janssen E, Shulman AI, Son MB, Hong S, Jones K, Ilowite NT, Cron RQ, Higgins GC (2011) Anakinra as first-line disease-modifying therapy in systemic juvenile idiopathic arthritis: report of forty-six patients from an international multicenter series. Arthritis Rheum 63:545–555CrossRefPubMed

30.

Ruperto N, Brunner HI, Quartier P, Constantin T, Wulffraat N, Horneff G, Brik R, McCann L, Kasapcopur O, Rutkowska-Sak L, Schneider R, Berkun Y, Calvo I, Erguven M, Goffin L, Hofer M, Kallinich T, Oliveira SK, Uziel Y, Viola S, Nistala K, Wouters C, Cimaz R, Ferrandiz MA, Flato B, Gamir ML, Kone-Paut I, Grom A, Magnusson B, Ozen S, Sztajnbok F, Lheritier K, Abrams K, Kim D, Martini A, Lovell DJ, PRINTO; PRCSG (2012) Two randomized trials of canakinumab in systemic juvenile idiopathic arthritis. N Engl J Med 367:2396–2406CrossRefPubMed

31.

Glaser RL, Goldbach-Mansky R (2008) The spectrum of monogenic autoinflammatory syndromes: understanding disease mechanisms and use of targeted therapies. Curr Allergy Asthma Rep 8:288–298PubMedCentralCrossRefPubMed

32.

Kuemmerle-Deschner JB, Hachulla E, Cartwright R, Hawkins PN, Tran TA, Bader-Meunier B, Hoyer J, Gattorno M, Gul A, Smith J, Leslie KS, Jimenez S, Morell-Dubois S, Davis N, Patel N, Widmer A, Preiss R, Lachmann HJ (2011) Two-year results from an open-label, multicentre, phase III study evaluating the safety and efficacy of canakinumab in patients with cryopyrin-associated periodic syndrome across different severity phenotypes. Ann Rheum Dis 70:2095–2102CrossRefPubMed

33.

Alpay N, Sumnu A, Caliskan Y, Yazici H, Turkmen A, Gul A (2012) Efficacy of anakinra treatment in a patient with colchicine-resistant familial Mediterranean fever. Rheumatol Int 32:3277–3279CrossRefPubMed

34.

Lane T, Gillmore JD, Wechalekar AD, Hawkins PN, Lachmann HJ (2015) Therapeutic blockade of interleukin-6 by tocilizumab in the management of AA amyloidosis and systemic autoinflammatory disorders: a case series and review of the literature. Clin Exp Rheumatol PMID: 26120866

35.

Moser C, Pohl G, Haslinger I, Knapp S, Rowczenio D, Russel T, Lachmann HJ, Lang U, Kovarik J (2009) Successful treatment of familial Mediterranean fever with Anakinra and outcome after renal transplantation. Nephrol Dial Transplant 24:676–678CrossRefPubMed

36.

Ozcakar ZB, Ozdel S, Yilmaz S, Kurt-Sukur ED, Ekim M, Yalcinkaya F (2014) Anti-IL-1 treatment in familial Mediterranean fever and related amyloidosis. Clin Rheumatol. doi:10.1007/s10067-014-2772-2

37.

Scarpioni R, Rigante D, Cantarini L, Ricardi M, Albertazzi V, Melfa L, Lazzaro A (2015) Renal involvement in secondary amyloidosis of Muckle-Wells syndrome: marked improvement of renal function and reduction of proteinuria after therapy with human anti-interleukin-1beta monoclonal antibody canakinumab. Clin Rheumatol 34:1311–1316CrossRefPubMed

38.

Stojanov S, Lapidus S, Chitkara P, Feder H, Salazar JC, Fleisher TA, Brown MR, Edwards KM, Ward MM, Colbert RA, Sun HW, Wood GM, Barham BK, Jones A, Aksentijevich I, Goldbach-Mansky R, Athreya B, Barron KS, Kastner DL (2011) Periodic fever, aphthous stomatitis, pharyngitis, and adenitis (PFAPA) is a disorder of innate immunity and Th1 activation responsive to IL-1 blockade. Proc Natl Acad Sci U S A 108:7148–7153PubMedCentralCrossRefPubMed

39.

Lane T, Loeffler JM, Rowczenio DM, Gilbertson JA, Bybee A, Russell TL, Gillmore JD, Wechalekar AD, Hawkins PN, Lachmann HJ (2013) AA amyloidosis complicating the hereditary periodic fever syndromes. Arthritis Rheum 65:1116–1121CrossRefPubMed

40.

Oner A, Erdogan O, Demircin G, Bulbul M, Memis L (2003) Efficacy of colchicine therapy in amyloid nephropathy of familial Mediterranean fever. Pediatr Nephrol 18:521–526PubMed

41.

Castano E, Palmer MB, Vigneault C, Luciano R, Wong S, Moeckel G (2015) Comparison of amyloid deposition in human kidney biopsies as predictor of poor patient outcome. BMC Nephrol 16:64PubMedCentralCrossRefPubMed

42.

Tennent GA, Lovat LB, Pepys MB (1995) Serum amyloid P component prevents proteolysis of the amyloid fibrils of Alzheimer disease and systemic amyloidosis. Proc Natl Acad Sci U S A 92:4299–4303PubMedCentralCrossRefPubMed

43.

Gillmore JD, Tennent GA, Hutchinson WL, Gallimore JR, Lachmann HJ, Goodman HJ, Offer M, Millar DJ, Petrie A, Hawkins PN, Pepys MB (2010) Sustained pharmacological depletion of serum amyloid P component in patients with systemic amyloidosis. Br J Haematol 148:760–767CrossRefPubMed

44.

Pepys MB, Herbert J, Hutchinson WL, Tennent GA, Lachmann HJ, Gallimore JR, Lovat LB, Bartfai T, Alanine A, Hertel C, Hoffmann T, Jakob-Roetne R, Norcross RD, Kemp JA, Yamamura K, Suzuki M, Taylor GW, Murray S, Thompson D, Purvis A, Kolstoe S, Wood SP, Hawkins PN (2002) Targeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosis. Nature 417:254–259CrossRefPubMed

45.

Bodin K, Ellmerich S, Kahan MC, Tennent GA, Loesch A, Gilbertson JA, Hutchinson WL, Mangione PP, Gallimore JR, Millar DJ, Minogue S, Dhillon AP, Taylor GW, Bradwell AR, Petrie A, Gillmore JD, Bellotti V, Botto M, Hawkins PN, Pepys MB (2010) Antibodies to human serum amyloid P component eliminate visceral amyloid deposits. Nature 468:93–97PubMedCentralCrossRefPubMed

46.

Pepys-Vered ME, Pepys MB (2014) Targeted treatment for amyloidosis. Isr Med Assoc J 16:277–280PubMed

47.

Westermark GT, Fändrich M, Westermark P (2014) AA amyloidosis: pathogenesis and targeted therapy. Annu Rev Pathol 10:321–344CrossRefPubMed

48.

Aganna E, Hawkins PN, Ozen S, Pettersson T, Bybee A, McKee SA, Lachmann HJ, Karenko L, Ranki A, Bakkaloglu A, Besbas N, Topaloglu R, Hoffman HM, Hitman GA, Woo P, McDermott MF (2004) Allelic variants in genes associated with hereditary periodic fever syndromes as susceptibility factors for reactive systemic AA amyloidosis. Genes Immun 5:289–293CrossRefPubMed

49.

Obici L, Raimondi S, Lavatelli F, Bellotti V, Merlini G (2009) Susceptibility to AA amyloidosis in rheumatic diseases: a critical overview. Arthritis Rheum 61:1435–1440CrossRefPubMed

50.

Cantarini L, Lucherini OM, Simonini G, Galeazzi M, Baldari CT, Cimaz R (2012) Systemic-onset juvenile idiopathic arthritis complicated by early onset amyloidosis in a patient carrying a mutation in the MEFV gene. Rheumatol Int 32:465–467CrossRefPubMed

Titel: Anti-interleukin 1 treatment in secondary amyloidosis associated with autoinflammatory diseases
verfasst von: Rezan Topaloglu
Ezgi Deniz Batu
Diclehan Orhan
Seza Ozen
Nesrin Besbas
Publikationsdatum: 01.04.2016
Verlag: Springer Berlin Heidelberg
Erschienen in: Pediatric Nephrology / Ausgabe 4/2016
Print ISSN: 0931-041X
Elektronische ISSN: 1432-198X
DOI: https://doi.org/10.1007/s00467-015-3249-5

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Anti-interleukin 1 treatment in secondary amyloidosis associated with autoinflammatory diseases

Abstract

Background

Methods

Results

Conclusions

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